Leber's Congenital Amaurosis
Leber's congenital amaurosis (LCA) is a rare genetic disorder that causes severe vision loss or blindness at birth or in early childhood due to retinal degeneration.
Leber Congenital Amaurosis: Sepofarsen Treatment Study
We are investigating a new treatment for people with Leber Congenital Amaurosis caused by a specific genetic mutation. The study will assess how well it improves vision and its safety over time.
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Are you at least 18 years old?
- Are you currently pregnant or breastfeeding?
- Do you have a history of glaucoma or high eye pressure (greater than 24 mmHg)?
- Are you currently taking any medications known to be toxic to the lens or retina, like Plaquenil or tamoxifen?
- Have you had any eye surgery in the past month?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Sepofarsen
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Universitair Ziekenhuis Gent
Ophthalmology
Ledeberg, Belgium
Centre Hospitalier National D'Ophtalmologie Quinze-Vingts
Centre de maladies rares CHNO des Quinze Vingt
Quinze-Vingts, France
Justus-Liebig-Universitaet Giessen
Klinik und Poliklinik für Augenheilkunde
Gießen, Germany
Sponsor: Laboratoires Thea
Last updated: Nov 27, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.